Arrakis Therapeutics Announces Exclusive License to RNA-targeted Small Molecule Technology from the University of Pennsylvania

Company continues to build leading technology portfolio for innovative targeting of RNA

Waltham, Mass., December 13, 2017 – Arrakis Therapeutics, a pioneering biopharmaceutical company, today announced that it has entered into an exclusive license agreement with the University of Pennsylvania to access intellectual property and technology related to small molecule drug candidates that directly target RNA. The license allows utilization of technology related to families of compounds that selectively bind specific nucleic acid structures, called three-way junctions, that are present in folded RNA and have attractive characteristics for potential therapeutic targeting of RNA. The technology was developed by David Chenoweth, Ph.D., Associate Professor in the Department of Chemistry at the University of Pennsylvania.

Emerging research has demonstrated that RNA folds into intricate three-dimensional shapes with predictable loops, bulges and pockets that can be targeted with rationally-designed small-molecule drugs. The ability to target RNA with small molecules opens up the potential to access the biological and potentially disease-relevant activity of more than 200,000 RNA transcripts, representing a vast number of therapeutic targets that are largely out of reach with today’s drug development tools. Arrakis was founded based on intellectual property and insights to target RNA with small molecules, and this license exemplifies the company’s strategy to integrate technologies and expertise in this emerging field.

“We are delighted to leverage the technology and insights of David Chenoweth, a leader in the emerging, interdisciplinary field of the biophysics of RNA to identify novel small molecules that bind RNA and modulate RNA function,” said Jennifer Petter, Ph.D., Founder and Chief Scientific Officer of Arrakis. “Exclusive access to this IP further positions Arrakis on the leading edge of targeting RNA with small molecules, as we access, create, and integrate technology to discover a powerful new medicines for human disease.”

At the University of Pennsylvania, Dr. Chenoweth holds appointments in the Chemistry Department in the School of Arts and Sciences, the Biochemistry and Molecular Biophysics Graduate Group in the Perelman School of Medicine, and the Bioengineering Graduate Group in the School of Engineering and Applied Science. Dr. Chenoweth serves as a member of the Scientific Advisory Board of Arrakis.

“The lifecycle of RNA is very complex and we are just beginning to understand and predict the different structural components that impact the production of proteins from mRNA,” said Dr. Chenoweth. “Our team has just begun to elucidate the role of three-way junctions and how they may open a new avenue for the discovery of small molecules that bind to RNA and intervene in disease processes.”

About RNA-targeted Small Molecules (rSMs)

Emerging insights and technologies focused on understanding the structure of RNA enable the design of RNA-targeted small molecules, or rSMs, a new class of medicines that directly bind and modify the biological function of RNA to treat disease. Unlike conventional drug discovery approaches that focus on proteins, an rSM is designed to modulate function of an individual RNA, and, in the case of mRNA, the expressed protein, by selectively binding to specific, predictable structures in functional regions in the RNA. This new drug discovery approach enables the design of small-molecule drugs that can unlock the therapeutic potential of well-known targets that are not accessible with today’s drugs, as well as identifying new drug targets to intervene in diseases in new ways.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. The company has developed a proprietary platform to identify new RNA targets and drug candidates to treat diseases unaddressed by today’s medicines. Arrakis is building a proprietary pipeline of RNA-targeted small molecules focused on neurologic diseases, cancer, and rare genetic diseases. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Mass. Please visit www.arrakistx.com.